Novo Nordisk receives FDA approval for Alhemo injection to treat hemophilia A and B in adults and adolescents aged 12 and older.

Novo Nordisk has received expanded FDA approval for Alhemo injection, a treatment to prevent or lessen bleeding episodes in adult and pediatric patients aged 12 and older with hemophilia A or B. The approval allows Alhemo to be used in both adult and pediatric patients, expanding its market potential.

Title: Novo Nordisk's Alhemo Injection Expands FDA Approval for Hemophilia Treatment

Novo Nordisk has received expanded FDA approval for Alhemo injection, a treatment designed to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 and older with hemophilia A or B. This approval allows Alhemo to be used in both adult and pediatric patients, significantly expanding its market potential.

The FDA approval is based on phase 3 trial data from the Explorer8 study, which demonstrated the safety and efficacy of Alhemo in patients with hemophilia A or B without inhibitors. The study showed a 79% reduction in annualized bleeding rate (ABR) in patients with hemophilia B (HB) without inhibitors and an 86% reduction in patients with hemophilia A (HA) without inhibitors using Alhemo prophylaxis compared to no prophylaxis [1].

Alhemo is a subcutaneous injectable pen that blocks a protein called tissue factor pathway inhibitor (TFPI), enhancing the production of thrombin, which helps clot the blood and prevent bleeding. This innovation provides a new treatment option for patients with hemophilia A or B without inhibitors, who previously relied on intravenous infusions [1].

The approval of Alhemo for patients without inhibitors builds on the December 2024 approval for those with inhibitors, further expanding the market for this treatment. The approval of Alhemo for both adult and pediatric patients with hemophilia A or B without inhibitors marks a significant step forward in addressing the unmet needs of this community [1].

The Explorer8 study, which evaluated the efficacy and safety of Alhemo, enrolled 156 males with hemophilia A or B without inhibitors. The study showed statistically significant reductions in ABR for both HB and HA patients, with an average ABR of 3.1 and 2.7, respectively, compared to 14.8 and 19.3, respectively, in the no prophylaxis group [1].

The most common adverse reactions reported in the study were injection site reactions and headache, both occurring in 7% of patients [1]. Alhemo is provided in prefilled, premixed pens for subcutaneous injection, offering a convenient and accessible treatment option for patients [1].

Novo Nordisk's Alhemo injection now offers a once-daily prophylaxis option for patients with hemophilia A or B without inhibitors, providing a new treatment choice that may help decrease bleeding rates and improve quality of life [1].

References
[1] https://pipelinereview.com/fda-approves-alhemo-as-once-daily-prophylactic-treatment-to-prevent-or-reduce-the-frequency-of-bleeding-episodes-for-adults-and-children-12-years-of-age-and-older-with-hemophilia-a-or-b-ha-hb/